TY - JOUR
T1 - A prospective, multicenter, randomized phase II study to evaluate the efficacy and safety of eculizumab in patients with guillain-barré syndrome (GBS)
T2 - Protocol of Japanese eculizumab trial for GBS (JET-GBS)
AU - JET-GBS Group
AU - Yamaguchi, Nobuko
AU - Misawa, Sonoko
AU - Sato, Yasunori
AU - Nagashima, Kengo
AU - Katayama, Kanako
AU - Sekiguchi, Yukari
AU - Iwai, Yuta
AU - Amino, Hiroshi
AU - Suichi, Tomoki
AU - Yokota, Takanori
AU - Nishida, Yoichiro
AU - Kohara, Nobuo
AU - Hirata, Koichi
AU - Nishiyama, Kazutoshi
AU - Yabe, Ichiro
AU - Kaida, Ken Ichi
AU - Suzuki, Norihiro
AU - Nodera, Hiroyuki
AU - Tsuji, Shoji
AU - Koike, Haruki
AU - Kira, Jun Ichi
AU - Hanaoka, Hideki
AU - Kusunoki, Susumu
AU - Kuwabara, Satoshi
N1 - Publisher Copyright:
© Nobuko Yamaguchi, Sonoko Misawa, Yasunori Sato, Kengo Nagashima, Kanako Katayama, Yukari Sekiguchi, Yuta Iwai, Hiroshi Amino, Tomoki Suichi, Takanori Yokota, Yoichiro Nishida, Nobuo Kohara, Koichi Hirata, Kazutoshi Nishiyama, Ichiro Yabe, Ken-Ichi Kaida, Norihiro Suzuki, Hiroyuki Nodera, Shoji Tsuji, Haruki Koike, Jun-Ichi Kira, Hideki Hanaoka, Susumu Kusunoki, Satoshi Kuwabara, JET-GBS Group. Originally published in JMIR Research Protocols (http://www.researchprotocols.org), 07.11.2016. This is an open-access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work, first published in JMIR Research Protocols, is properly cited. The complete bibliographic information, a link to the original publication on http://www.researchprotocols.org, as well as this copyright and license information must be included.
PY - 2016/10
Y1 - 2016/10
N2 - Background: Guillain-Barré syndrome (GBS) is an immune-mediated neuropathy that causes acute flaccid paralysis. Immunoglobulin and plasma exchange are established treatments for GBS; however, a substantial number of patients, particularly those with severe disease, have poor recovery and residual deficits. Recent studies suggest that complement activation plays a pivotal role in GBS-associated axonal degeneration, and eculizumab is a humanized monoclonal antibody that specifically binds to complement component 5 and potently inhibits complement activation. Objective: This clinical trial aims to evaluate the efficacy and safety of eculizumab, a humanized monoclonal antibody directed against complement component 5, for treatment of GBS. Methods: The Japanese Eculizumab Trial for GBS (JET-GBS) is a prospective, multicenter, placebo-controlled, double-blind, randomized phase II study conducted at 13 tertiary neurology centers and is funded by the Japan Agency for Medical Research and Development. A total of 33 GBS patients unable to walk independently within 2 weeks from symptom onset (Hughes functional grade 3-5) were randomized at a 2:1 ratio to receive either intravenous eculizumab (900 mg/day) or placebo once weekly for 4 weeks, followed by 20 weeks of follow-up. The primary endpoint for efficacy is the proportion of patients who regain their ability to walk without aid at 4 weeks after the first dose of the study treatment, while primary safety outcomes are the incidence of adverse events and serious adverse events during the trial. Results: Enrollment for the trial began in August 2015. This trial is still ongoing. All participants have been enrolled, and follow-up will be completed in October 2016. Conclusions: This study is the first to investigate the efficacy and safety of eculizumab for GBS. In case of a positive result, we will plan a phase III trial to investigate this issue in a larger number of patients.
AB - Background: Guillain-Barré syndrome (GBS) is an immune-mediated neuropathy that causes acute flaccid paralysis. Immunoglobulin and plasma exchange are established treatments for GBS; however, a substantial number of patients, particularly those with severe disease, have poor recovery and residual deficits. Recent studies suggest that complement activation plays a pivotal role in GBS-associated axonal degeneration, and eculizumab is a humanized monoclonal antibody that specifically binds to complement component 5 and potently inhibits complement activation. Objective: This clinical trial aims to evaluate the efficacy and safety of eculizumab, a humanized monoclonal antibody directed against complement component 5, for treatment of GBS. Methods: The Japanese Eculizumab Trial for GBS (JET-GBS) is a prospective, multicenter, placebo-controlled, double-blind, randomized phase II study conducted at 13 tertiary neurology centers and is funded by the Japan Agency for Medical Research and Development. A total of 33 GBS patients unable to walk independently within 2 weeks from symptom onset (Hughes functional grade 3-5) were randomized at a 2:1 ratio to receive either intravenous eculizumab (900 mg/day) or placebo once weekly for 4 weeks, followed by 20 weeks of follow-up. The primary endpoint for efficacy is the proportion of patients who regain their ability to walk without aid at 4 weeks after the first dose of the study treatment, while primary safety outcomes are the incidence of adverse events and serious adverse events during the trial. Results: Enrollment for the trial began in August 2015. This trial is still ongoing. All participants have been enrolled, and follow-up will be completed in October 2016. Conclusions: This study is the first to investigate the efficacy and safety of eculizumab for GBS. In case of a positive result, we will plan a phase III trial to investigate this issue in a larger number of patients.
KW - Antiganglioside antibody
KW - Clinical trial
KW - Complement activation
KW - Eculizumab
KW - Guillain-Barré syndrome
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U2 - 10.2196/resprot.6610
DO - 10.2196/resprot.6610
M3 - Article
AN - SCOPUS:85014083861
VL - 5
JO - JMIR Research Protocols
JF - JMIR Research Protocols
SN - 1929-0748
IS - 4
M1 - e210
ER -